US RNA-targeted drug developer Ionis Pharmaceuticals (Nasdaq: IONS) has announced positive results from a Phase II study of ION224, an investigational DGAT2 antisense inhibitor in development for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), previously referred to as non-alcoholic steatohepatitis (NASH).
The study met its primary endpoint at both doses (120mg and 90mg), achieving liver histologic improvement, and also met the important secondary endpoint of MASH resolution, and the news sent Ionis’ shares up 7% in pre-market trading on Wednesday.
The results come as companies race to bring the first approved treatment for the disease and tap into a global market expected to surpass $16 billion by 2030, according to market research firm Vision Research Reports. So far, although a number of drugmakers are the therapy area, none have won regulatory approval.
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